Pivekimab Sunirine in Blastic Plasmacytoid Dendritic Cell Neoplasm

Area of Science:

• Hematology
• Oncology
• Immunotherapy

Background:

• Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare myeloid malignancy.
• BPDCN is characterized by CD123 overexpression and typically has a poor prognosis.

Purpose of the Study:

• To evaluate the efficacy and safety of pivekimab sunirine (PVEK) in adults with BPDCN.
• PVEK is a novel antibody-drug conjugate targeting CD123.

Main Methods:

• A phase I/II, open-label, multicenter study.
• Adults with frontline or relapsed/refractory BPDCN received PVEK at 0.045 mg/kg every 3 weeks.
• The primary endpoint was composite complete response (CCR) rate in the primary analysis population (PAP) of frontline de novo BPDCN.

Main Results:

• In the PAP (n=20), the CCR rate was 75%, with a median overall survival (OS) of 16.6 months.
• For relapsed/refractory BPDCN (n=51), the CCR rate was 14%, with a median OS of 5.8 months.
• Common adverse events included peripheral edema and fatigue; serious events included neutropenia and febrile neutropenia. Veno-occlusive disease (VOD) was observed in some patients, particularly those undergoing stem-cell transplant (SCT).

Conclusions:

• PVEK demonstrated high and durable responses, particularly in frontline BPDCN.
• The antibody-drug conjugate has a manageable safety profile with convenient dosing.
• PVEK represents a promising therapeutic option for BPDCN.